Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO

Non Interventional Longitudinal Study To Assess The Acceptability And The Satisfaction Of Patients With Haemophilia A Treated Both On Demand And On Prophylaxis With A New Factor Viii Delivery System Fusengo

This study aims to investigate prospectively throughout a period of 3- 6 months and not to exceed 12 months (according to local therapeutic plans) patients experiences of treatment with Factor VIII in the new device named FuseNGO, a new delivery system for Factor VIII compared to their previous delivery system.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Device: Refacto FusENGO

• Study Type: Observational
• Enrollment (Actual): 86

Contacts and Locations

Study Locations

• Italy
  • Bari, Italy, 70121
    • Azienda Ospedale Policlinico Consorziale
  • Cagliari, Italy, 09100
    • Struttura Complessa Di Oncoematologia Pediatrica E Patologia Della Coagulzione
  • Catania, Italy, 95124
    • Ospedale Ferrarrotto
  • Catanzaro, Italy, 88100
    • Azienda Ospedaliera Ciaccio - Ospedale Pugliese
  • Firenze, Italy, 50134
    • Azienda Ospedaliera Careggi
  • Macerata, Italy, 62100
    • Centro regionale di diagnosi e trattamento dell'Emofilia e delle malattie dell'emostasi e trombosi
  • Milano, Italy
    • Ospedale Maggiore Policlinico Mangiagalli e Regina Elena
  • Napoli, Italy, 80131
    • Dipartimento Medicina Clinica e Sperimentale
  • Napoli, Italy, 81044
    • Centro Emofilia e Trombosi - Centro delle Microcitemie
  • Padova, Italy, 35128
    • Azienda Ospedaliera di Padova
  • Palermo, Italy, 90100
    • Policlinico P Giaccone
  • Reggio Emilia, Italy, 42100
    • Arcispedale S. Maria Nuova, AO di Reggio Emilia
  • Rome, Italy
    • Policlinico Agostino Gemelli
  • Torino, Italy, 10126
    • Ospedale Molinette
  • Torino, Italy
    • SC Servizio Immunotrasfusionale
  • Udine, Italy, 33100
    • SOS Malattie Emorragiche e Trombotiche AOU "S.M.M." Udine
  • Verona, Italy, 37126
    • Anna Chiara Giuffrida
  • Lazio
    • Roma, Lazio, Italy, 00161
      • Istituto di Ematologia
  • Sardegna
    • Sassari, Sardegna, Italy, 07100
      • Ospedale SS. Annunziata - USL 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

Approximately 200 patients with Hemophilia A (all severity levels) will be enrolled in approximately 20 centers in Italy.

Description

Inclusion Criteria:

• Male adults (aged 18 to 65 years) with Haemophilia A (all severity levels) who currently use FVIII treatment either prophylactically or on-demand using traditional factor VIII delivery mechanisms.
• Patients advised for any reason by their physician to switch to a new factor VIII delivery device, namely FuseNGO, before receiving any details about this study or Patients who requested to be treated with a new factor VIII delivery device namely FuseNGO, before receiving any details about this study.

Exclusion Criteria:

• Patients not previously recommended by their physician to switch to this new factor VIII delivery system or Patients who did not previously ask their physician to be switched to this new factor VIII delivery system.
• Patients for which it is anticipated that 10 infusions will not occur in the 12 months following their inclusion in the study.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Arm 1: Device	Device: Refacto FusENGO; Dosage will be chosen by PI based on patient condition.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ease of Using Clotting Factor Treatment Score	HaemoPREF is a participant rated 14-item instrument to measure experience of clotting factor treatment including ease of use, burden, impact, risk and influence of others on treatment choices. For determining score for ease of using clotting factor treatment, 4 items assessed by participants were: 1) ease to prepare treatment for injection, 2) ease to store treatment, 3) ease to dispose of containers, syringe and needle once used and 4) ease to use current treatment. Each of the 4 items was scored on a scale of 0 (not at all easy) to 10 (extremely easy), and were summed up, to give a total overall score range of 0 (no ease) to 40 (maximum ease). Higher scores indicate greater ease in using clotting factor treatment.	Final Visit (Month 8)
Time for Reconstructing the Drug	In this outcome measure time consumed for performing steps to reconstitute the drug prior to infusion of drug is reported.	Final Visit (Month 8)
Burden of Clotting Factor Treatment Score	HaemoPREF is a participant rated 14-item instrument to measure experience of clotting factor treatment including ease of use, burden, impact, risk and influence of others on treatment choices. For determining the score for burden of clotting factor treatment, 2 items were assessed by participants: 1) time consumption to treat with treatment and 2) difficulty in finding vein to inject treatment in to. Each of the 2 items was scored on a scale of 0 (most difficult) to 20 ( least difficult) and summed up to give a total overall score range of 0 (most burdened) to 40 (least burdened). Higher scores indicate a lower burden of treatment.	Final Visit (Month 8)
Impact of Clotting Factor Treatment Score	HaemoPREF is a participant rated 14-item instrument to measure experience of clotting factor treatment including ease of use, burden, impact, risk and influence of others on treatment choices. For determining score for impact of clotting factor treatment, 3 items were assessed by participants: 1) difficulty to travel for holidays or works, 2) difficulty to perform daily activities including work or study and 3) difficulty to perform social or leisure activities. Each of the 3 items was scored on a scale of 0 (most challenging) to 10 (least challenging), and summed up to give a total overall score range of 0 (greatest negative impact) to 30 (least negative impact). Higher scores indicate less negative impact on daily life.	Final Visit (Month 8)
Risk Associated With Clotting Factor Treatment Score	HaemoPREF is a participant rated 14-item instrument to measure experience of clotting factor treatment including ease of use, burden, impact, risk and influence of others on treatment choices. For determining score for risk associated with clotting factor treatment, 3 items were assessed by participants: 1) worried about getting infected with other disease while using the treatment, 2) worried to contaminate the treatment while preparing for injection and 3) worried to inject treatment by own. Each of the 3 items was scored on a scale of 0 (most concerned) to 10 (not at all concerned), and summed up to give a total overall score range of 0 (most worried) to 30 (least worried). Higher scores indicate lower levels of worry associated with treatment.	Final Visit (Month 8)
Overall Satisfaction Score With Refacto AF FuseNGO	HaemoPREF: participant rated 14-item instrument to measure experience of clotting factor treatment. 14 items: 1) ease to prepare treatment for injection, 2) ease to store treatment, 3) ease to dispose container, syringe, needle once used and 4) ease to use treatment, 5) time consumed with treatment, 6) difficulty in finding a vein to inject treatment, 7) difficulty to travel, 8) difficulty to do daily activities, 9) difficulty to do social or leisure activities, 10) worried for getting infected with other disease while using the treatment, 11) worried to contaminate the treatment while preparing for injection, 12) worried to inject by own, 13) importance of family's opinion to use treatment, 14) importance what others use for their hemophilia. Each item was scaled from 0 (no satisfaction) to 10 (maximum satisfaction). Overall satisfaction score was the sum of 14 items, ranged from 0 (no satisfaction) to 140 (maximum satisfaction). Higher scores indicate greater treatment satisfaction.	Final Visit (Month 8)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Events and Serious Adverse Events	An adverse event (AE) was any untoward medical occurrence in a participant who received treatment using Refacto AF Fusnego without regard to possibility of causal relationship. Serious adverse event was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both serious and non-serious AEs.	Baseline up to 28 days after last dose of drug (up to Month 12)

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): January 27, 2014
• Primary Completion (Actual): December 12, 2016
• Study Completion (Actual): December 12, 2016

Study Registration Dates

• First Submitted: October 8, 2013
• First Submitted That Met QC Criteria: October 9, 2013
• First Posted (Estimate): October 10, 2013

Study Record Updates

• Last Update Posted (Actual): September 25, 2018
• Last Update Submitted That Met QC Criteria: September 24, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII
• Other Study ID Numbers: B1831081